CN108138159A - 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途 - Google Patents

经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途 Download PDF

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CN108138159A
CN108138159A CN201680043108.6A CN201680043108A CN108138159A CN 108138159 A CN108138159 A CN 108138159A CN 201680043108 A CN201680043108 A CN 201680043108A CN 108138159 A CN108138159 A CN 108138159A
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sequence
aav
nucleic acid
encoding human
albumen
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凯瑟琳·A·海
泽维尔·安谷拉
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Childrens Hospital of Philadelphia CHOP
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    • A61K38/36Blood coagulation or fibrinolysis factors
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    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482Serine endopeptidases (3.4.21)
    • A61K38/4846Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
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CN201680043108.6A 2015-06-23 2016-06-23 经修饰的因子ix、以及用于基因转移到细胞、器官和组织的组合物、方法和用途 Pending CN108138159A (zh)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201562183599P 2015-06-23 2015-06-23
US62/183,599 2015-06-23
US201662315453P 2016-03-30 2016-03-30
US62/315,453 2016-03-30
US201662338315P 2016-05-18 2016-05-18
US62/338,315 2016-05-18
US201662348781P 2016-06-10 2016-06-10
US62/348,781 2016-06-10
US201662349572P 2016-06-13 2016-06-13
US62/349,572 2016-06-13
PCT/US2016/039075 WO2016210170A1 (en) 2015-06-23 2016-06-23 Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues

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EP (2) EP3313991A4 (es)
JP (3) JP6831779B2 (es)
KR (2) KR102178322B1 (es)
CN (1) CN108138159A (es)
AU (2) AU2016282781A1 (es)
BR (1) BR112017027956A2 (es)
CA (1) CA2990193A1 (es)
CO (1) CO2018000134A2 (es)
HK (1) HK1252351A1 (es)
IL (2) IL309741A (es)
MX (2) MX2018000307A (es)
PE (1) PE20180675A1 (es)
PH (1) PH12017502424A1 (es)
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CN111647625A (zh) * 2019-12-25 2020-09-11 深圳三智医学科技有限公司 一种提高人凝血因子ix表达水平的方法
CN112739818A (zh) * 2018-08-20 2021-04-30 Ucl商业有限责任公司 编码因子ix的核苷酸
CN112955174A (zh) * 2018-07-09 2021-06-11 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
CN113817759A (zh) * 2020-07-10 2021-12-21 南京吉迈生物技术有限公司 修饰的因子ix、组合物、方法及其在基因治疗中的应用
CN114302968A (zh) * 2019-08-29 2022-04-08 美国西门子医学诊断股份有限公司 用于检测aav脱落的试剂和方法
CN115461465A (zh) * 2020-04-07 2022-12-09 密苏里大学管理机构 用于AAV基因治疗的无CpG的ITR
WO2022268110A1 (zh) * 2021-06-23 2022-12-29 英斯培瑞有限公司 用于治疗b型血友病的组合物和方法
WO2023202469A1 (zh) * 2022-04-19 2023-10-26 康霖生物科技(杭州)有限公司 一种用于遗传性凝血因子缺乏病治疗的核酸构建体及其用途

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